a study to investigate lp352 in children and adults with developmental and epileptic encephalopathies (dee)

Conditions: Developmental and Epileptic Encephalopathy

Summary: this (deep ocean study) is a double-blind, randomized, placebo-controlled, multicenter study to investigate the efficacy, safety, and tolerability of lp352 in the treatment of seizures in children and adults with dee. the study consists of 3 main phases: screening, titration period, maintenance period, followed by a taper period and follow-up. the total duration of the study will be approximately 24 months.


Eligibility criteria:
inclusion criteria: * participants who are characterized as having lennox-gastaut syndrome (lgs) must fulfill all of the following criteria: * onset of seizures at ≤8 years old * history of tonic/tonic-atonic seizures plus at least 1 of the following seizure type(s): atypical absence, atonic, myoclonic, focal impaired awareness, generalized tonic-clonic, nonconvulsive status epilepticus, or epileptic spasms * presence of developmental plateauing or regression * history of electroencephalogram (eeg) showing generalized slow (\<2.5 hertz \[hz\]) spike-and-wave complexes * participants who are characterized as having dee (other) must fulfill all of the following criteria: * does not meet criteria for lgs * onset of seizures at ≤5 years old * presence of developmental plateauing or regression * history of multiple seizure types * history of interictal eeg background showing diffuse or multifocal slowing (with or without epileptiform activity) * the participant has a current occurrence of at least 1 of the following countable motor seizure types: generalized tonic-clonic, tonic (bilateral), clonic (bilateral), atonic (bilateral) with truncal/leg involvement, focal motor (including hemiclonic), and focal to bilateral tonic-clonic. * the participant has demonstrated an average of at least 4 countable motor seizures per month for each of the 3 months prior to screening. * the participant has been taking 1 to 4 antiseizure medications (asms) at a stable dose for at least 4 weeks prior to screening. * the participant, parent, or caregiver is willing and able (in the judgment of the investigator) to comply with completion of the diaries throughout the study. * the participant must be willing and able to provide written informed consent; in instances where the participant is unable to provide consent, an appropriate legal representative. exclusion criteria: * the participant has a diagnosis of dravet syndrome (ds) or has a mutation of the sodium channel protein type 1 subunit alpha (scn1a) gene consistent with ds. * the participant has been admitted to a medical facility for treatment of status epilepticus requiring mechanical ventilation within 3 months prior to screening. * the participant has a neurodegenerative disorder as indicated by magnetic resonance imaging or genetic testing. * the participant has an acquired lesion/injury unrelated to the primary etiology that could contribute as a secondary cause of seizures. * the participant is receiving exclusionary medications. * the participant has used of any cannabis product or cannabidiol that is not in oral solution/capsule/tablet form, not obtained from a government-approved dispensary, or contains ≥50% delta-9-tetrahydrocannabinol (thc). * the participant has unstable, clinically significant neurologic (other than the disease being studied; eg, recurrent strokes), psychiatric, cardiovascular (eg, pulmonary arterial hypertension, cardiac valvulopathy, orthostatic hypotension/tachycardia), pulmonary, hepatic, renal, metabolic, gastrointestinal, urologic, immunologic, hematopoietic, or endocrine disease or other abnormality which may impact the ability of the participant to participate or potentially confound the study results. * the participant is unable or unwilling to comply with any of the study requirements or timelines.

Keywords: developmental and epileptic encephalopathy lp352 seizures bexicaserin antiseizure medications (asms) epilepsy lennox-gastaut syndrome (lgs) neurodevelopmental disorders deep ocean